Coming Together to Ensure Neurological Treatment Access: Q&A With Kavita V. Nair, PhD

Am J Manag Care. 2025;31(Spec. No. 3):SP150-SP152. https://doi.org/10.37765/ajmc.2025.89709

PHEO: What led you to specialize in the intersection of neurology and pharmaceutical outcomes research?

NAIR: Most great things in life, you just come upon by chance. I’ve been an outcomes researcher for 27 years, and about 13 or 14 years ago, the innovation in neurology that we have now just didn’t quite exist…. In 2010, multiple sclerosis [MS] had its first oral drug, fingolimod [Gilenya], [approved]. I happened to meet with one of my mentors, Timothy Vollmer, MD, who’s now retired, and we started talking about things that were happening in the outcomes world, or comparative effectiveness research…. Neurology just didn’t have a lot of new drugs. Now we do, but they just didn’t exist [then]. Until that point, MS had only 4 drugs, many of them several decades old, and this new drug was oral.... We had this conversation about how you don’t always need to have clinical trials to compare drugs, and companies aren’t just going to compare head-to-head trials because the FDA doesn’t require the gold standard.

Kavita V. Nair, PhD

That concept—you always have to have someone champion for you, and then you have to champion for someone else—led to this conversation with the neuroimmunology group in the Department of Neurology about “How can we look at our data?” We have a large MS center of excellence at CU that has more than 4000 patients. It gave us a chance to look at these newer drugs—at the time, this was a newer drug class, a newer disease-modifying therapy, that was highly efficacious compared with some of the older drugs—and design a real-world evidence study and compare within a population. That kicked off looking at how we can do this in neurology.

Then in MS, fast forward to 2025, and we went from having 4 disease-modifying therapies that are FDA approved to more than 22 drugs and counting, so we were able to work together and create this framework of what we want to know. Neurologists and patients are presented with several options, which is very exciting, but how do you choose? How do you have evidence when the drugs’ markets and availability are rapidly evolving? That [led to] the intersection of: How do you compare drugs? How do you do it in a way that the drugs and the populations are comparable? How do you use methods we’ve developed in outcomes research, and how do you apply them? How do you use data sources that aren’t some of the more traditional data sources, such as claims? How do you [handle] the intersectionality of [electronic health] records and claims? How do you try to incorporate some patient-reported outcomes? How do you incorporate imaging measures? They’re all parts of the same concept of comparative effectiveness research.

And that grew, and then today, several years later, we’re applying the same concepts to other disease states in neurology where newer drugs have [been developed], as well in some rare neurological disease states, and now, of course, with Alzheimer disease, the same concepts, newer methods, and larger and more integrated data sources as well.

PHEO: What a journey!

NAIR: That’s right, and thankfully, for the patients, a great journey that keeps evolving.

PHEO: Could you tell me about your typical work day at CU Anschutz’s Center for Pharmaceutical Outcomes Research and Department of Neurology?

NAIR: I spend the majority of my time within the Department of Neurology, but we have different aspects. I bring the partnerships from industry to the Department of Neurology. With newer drugs, even just understanding that there can be really good synergistic partnerships between the industry and neurology was fairly new to some of the subspecialties in neurology—so with MS, with 22 drugs, the partnership was a lot easier. One aspect of my day, or my job, if you will, is working with industry and [neurology] to talk about: How do we design real-world studies or pragmatic trials, and where are those synergies? What is the unmet need right now? Yes, it is sometimes comparing different kinds of drugs. But in the past few years, it’s looking at other things. We have different populations within Colorado. So we have CU as the academic center, and as with most academic centers, all the subspecialties and the experts tend to be at the academic centers, but we have other sites of care that primarily see an underserved population, a Medicaid population. Regarding the availability of these FDA-approved drugs, for not only MS but also for Alzheimer disease or rare neurological diseases, they are FDA approved but tend to have a little bit of a sticker price shock for everyone. Are there differences in patients who come to the university and have the resources and the payer aspects to get those drugs? Are there disparities in who gets those drugs based on their payer type? More importantly, does it affect outcomes? That’s an aspect that everyone’s interested in. So that’s one part of my day, getting stakeholders together with a common interest and putting those studies together…. It’s like herding cats sometimes. It’s putting the idea together and taking it from soup to nuts.

Another part of my day…is from a policy standpoint. So much of what neurologists face—and actually all disciplines face it, but more so neurologists because of the explosion of drugs—is payer policies. One of the things I want to emphasize is I do work at different levels within the American Academy of Neurology, which is the largest professional organization for neurologists. It has more than 35,000 members, and I am in leadership positions in different groups. One of the groups I lead is the only one that works with payers. My goal is to make that relationship with payers one that is not adversarial and always finger pointing. You know, we have to do this together.

The other aspect is very policy-based, where we work with payers, sometimes in tandem and sometimes at the legislative level. I work a lot with the Colorado judiciary and the legislative group in different policy aspects to reform drug pricing policies. One of the efforts that I’m very involved in is gold-carding policies, which even payers are interested in, which asks: Is there a way to simplify prior authorization? For example, you’re coming to see a neurologist to get an MRI. Do we really have to have prior authorization if the neurologist orders the MRI? There are some common-sense solutions where you can get providers and payers together. We call it payvider. From a policy perspective, I’m actively involved in trying to conscript those policies and take them all the way to the legislative aspect, help craft bills, and maybe testify. I also encourage the neurology community to get involved at the state level because it really does matter. So that’s the second part of my day, if you will.

And then the third part of my day is encouraging the younger community, the next generation, because when you are being trained and receiving a lot of information, you need to have a sense of awareness about the health care system. Every clinician wants nothing more than to spend time with their patients, and everyone’s really committed to taking care of their patients, and that’s wonderful. But when pen hits the paper, so to speak, and you’re writing that prescription, how much a drug costs, the factors that go into the price of a drug, awareness of the drug pricing system—you don’t have to know exactly what a drug costs—[being aware of] those can help a patient even when they get access to a drug. I do didactics for neurology fellows. My first question, and it’s not meant to be a trick question, is, “If you were asked, what’s the price of drug X?” The right answer is: It really depends. If the drug is physician-administered, if the drug is a self-injectable, if it is an oral drug—it all depends on where the drug flows from the drug distribution chain. The pricing is very different. To share the basics of this, I teach a course or different variations of the course. I think that’s crucial for neurologists to understand. These are generally a few of the different buckets; I’m always reaching out to the stakeholders to say we have to work together as one.

PHEO: No two days are the same.

NAIR: Yes, and then some days I get to talk about what I do, and so that’s fun, too.

PHEO: What is an initiative that your team is working on that you’re passionate about?

NAIR: Excellent question. There are a couple things. One is that for the first time—and I’ve been on this campus as an academic for 27 years—we are able to really form partnerships. At CU, we have 3 hospital systems, and then we also have our largest disproportionate share hospital, Denver Health. Primarily, the patients who go to Denver Health are our indigent-care patients or those on Colorado Medicaid. And in the past year or so, for the first time, we’ve been able to really partner with them to look at how neurological care is being delivered. Why I’m so excited about that initiative is that some of the providers in neurology at CU Anschutz or our central campus are also the same providers who staff clinics at Denver Health, so you have this great natural experiment where you have the same providers but different populations [and] different insurers, and you can really look at any impact of drug pricing policies or drug coverage policies on outcomes, with everything else being very similar…. Colorado is an opt-in state, so we have a large Colorado Medicaid population. To be able to really look at how drug policies and coverage policies affect outcomes in this kind of a natural experiment, and to be able to partner with Denver Health and get through all the paperwork, I’m very excited about that. Now we can really examine, if there are any gaps, where they are, and if people are falling through the loopholes or the cracks, who they are and where we can intervene. Academic research is great—I’ve done 27 years of it—but we are at a point where we can actually look at research in a way in which we can identify care.

In Colorado, we’re very lucky to have different kinds of data that we can integrate. Typically, when you think of outcomes research, you think of a secondary database, but we are able to integrate several different data sources that include the electronic health record, [but also] the ability to include claims data for all of Colorado, so you can follow a patient. We can look at different indices that give us clusters of geographical, sociodemographic, or social determinants of health data. So we’re putting years and years of [data together that] I wished we had when I was a graduate student to get a holistic picture to identify the patients who are falling through the cracks and what we need to do and work together on. I’m truly excited about that.

The other thing we’re really excited about is that for the first time, we have this controversy: We’re starting to have drugs [for patients with] Alzheimer disease, and we’re going to have more. That dam has burst, and we are now having to figure out pathways to treat those patients. There might be different schools of thought on how effective these drugs are, but this has just opened the floodgates. This is what the MS [field] was [experiencing] years ago, and we are now, again, taking the clinical piece and figuring out how to treat the number of patients when this is not a rare neurological disease. We don’t have as many behavioral neurologists as we need, so how do we figure this out? Everyone is touched by Alzheimer disease and related dementias in some fashion. So, how do we take this increasing demand, with drugs we have now that have some benefit—people may disagree on what that level is—with other drugs coming in the pipeline? How do we see, treat, diagnose, and manage this largest segment of our population? It’s not just the folks [older than] 75 years; it’s Gen X. It’s me; so it’s very challenging, very exciting, very personal. My father passed away from Alzheimer disease 2 years ago. I am [in the] potential age demographic. It’s exciting and challenging all at the same time.

PHEO: I can see why you’re passionate about that. Are there any novel avenues of research on neurological treatments that you’re excited to investigate?

NAIR: As I mentioned, in Alzheimer disease, there are new mechanisms of action. Again, I’m a health outcomes researcher, so I leave that to the bench scientists, but we know in health services research that if you can’t get access to a drug, then it just sits on a shelf. So, how do we get patients who can come from anywhere—from primary care or someone just sitting at home who feels like their partner or their parent [needs care]—and how do we wrap our heads around the problem? And it’s not just one center. We are really coalescing around other centers, and everyone is coming together to try to figure out best practices. That’s about as close as you can get to making an impact in people’s lives, so I’m very excited about that.

The other aspect is, for the first time now, we’re seeing CAR [chimeric antigen receptor] T-cell and gene therapies, which really seemed like something that in my lifetime [would be only] for other things, rare things, things we never hear about. But we are seeing it in some diseases like sickle cell anemia. We’re starting to see it in some neurological diseases like myasthenia gravis. The process—I’m oversimplifying this from a nonclinical standpoint—the essence is that it’s sort of one and done. If a patient qualifies for cell and gene therapy, they get one, and then they potentially do not require treatment ever again. This is happening now, all these discussions and all this angst, legitimately so, even for payers like Medicaid, which in Colorado covers some of these therapies—not for neurology yet, because we’re still in the early stages, but there are several neurological diseases where cell and gene therapy are being evaluated in different phases. I’m very excited about the fact that the therapeutic advances in neurology are in that area. This is maybe just my [idea of] utopia, but even payers like Medicaid are saying that if we cover it—and it’s not for everyone, it’s for the highest utilizers, it’s for the most refractory patients—that could that put an end to some extent to questions like: “Why is it so expensive?” That’s all we hear. Every headline about a new drug, a new neurological drug, even for babies with SMA [spinal muscular atrophy], it’s all the headlines. Could this actually turn the tide, not only for the patient and the prognosis, but even have the larger community thinking about value?

In our world, we collectively think about value assessment, and it’s been tough to get that concept, because I think people get stuck on, “This costs half a million dollars. This drug costs $1.2 million”—rightfully so, when resources are so limited. I’m very excited about what that could look like, and when it starts becoming more of a reality, how we could collectively start assessing what that looks like. It remains to be seen, but I think that could be within our horizon, so I’m very, very excited about what that could mean for the neurological community, obviously, and for patients, first and foremost. It’s within our grasp, and payers are starting to embrace it too because they’re realizing [it could be] one and done to deal with it, potentially.

PHEO: That’s exciting. We always cover them in the context of oncology or hematology.

NAIR: And in oncology and hematology, it’s lifesaving, right? This is more that it’s a chronic condition that doesn’t have to be chronic. And again, these are very oversimplified terms, but with neurology, we hear “That’s too expensive,” so we have step edits, and [patients need to not succeed on] 2 drugs. But what if you just didn’t have to live with the chronic condition?

PHEO: You touched on this earlier, but how can investigators and industry partners better collaborate to bring these neurology research insights to the patients in need?

NAIR: The collaborative relationship needs to go a little bit beyond, “Hey, we have a clinical trial; we need a PI [principal investigator].” I know that’s the basis. We need to expand beyond that because we need to be able to have researchers and industry come together and talk about all kinds of collaborative relationships. Those would be relationships when we’re looking at a real-world study, or what is the need among the patient community, once a drug is approved or as you’re waiting for the drug to be approved. You can’t change what you don’t assess. The Holy Grail is when we’ve decided that this chemical entity will be [investigated in] a clinical trial and we’re recruiting for phase 1, phase 2, phase 3. We need investigators; that’s the road well traveled. But I think if researchers and industry come together and start partnerships with a wider variety of researchers.… We love giants in our field. We love our mentors. But if you really want to know what patients feel and how to communicate, you need to get researchers who look like the patients they’re treating. You need to get a wider range of researchers of different ages, who look differently, who have different experiences, because that’s how you really connect to who the patients are.

Colorado is an interesting state because we are very metropolitan. Denver International Airport is one of the top 5 busiest airports in the US. I’ve been here 27 years, and if you ask anyone my age to go downtown, we all groan because the traffic is crazy. We have all the trappings of big city life, but we also have a huge rural component. We are the Wild West. We have 64 counties in Colorado; 5 are very densely populated and have all the hospitals and neurological care, but the rest of Colorado is very rural. You have neurology, but you don’t necessarily have neurology subspecialty. As you get deeper into the rural areas of Colorado, you just don’t have infusion centers, you don’t have that kind of subspecialty. When we think about partnerships and trying to find out what patients want, it is about differences in the way patients are and where they come from, but it’s also where they live. And so we need to start thinking outside the box and partnering industry and researchers on these differences.

If you’ve seen maps of America, America has a lot of land. There are people who live in areas where there are neurological deserts. For us to come together—I say this as an academic researcher—we have a responsibility to understand and think and put forward solutions. We need to start thinking outside the box and coming together for solutions that aren’t just one solution. “I want to get this drug approved” is noble; we have Alzheimer drugs that we didn’t before. But if you can’t get them to people who all have an equal probability or the best possibility of [benefiting from] the drug, then that’s only half of the equation. So come forth, have conversations, go to those areas where you have 1 neurologist who sees all kinds of specialties. They see [patients with] MS and Alzheimer and myasthenia gravis and epilepsy. Those are the patients that they have, and what does that look like?

PHEO: Was there anything that you wanted to add?

NAIR: If there was a takeaway, for us as a neurology community, the broader neurology community, for everyone to move forward and capture the essence of how we can do this, we have to come together: A neurologist, a payer, a PBM [pharmacy benefit manager], the pharmaceutical industry all have to come together. Neurology is mostly a noninterventional field; we don’t do procedures. We treat through drugs. The starting point is the pharmaceutical industry. That’s our unique health care system, and it’s very complicated. Our drug distribution chain and everything in between has payers, whether it’s specialty pharmacy payers, PBMs, all of the above, and pharmacy can be mail order, and then you have neurologist and patient, and so any picture [of the drug distribution chain], you’ll see it in different versions, because that’s the reality. For a practitioner or clinician to effectively prescribe drugs, for drugs to be accessible to the patient—a patient just doesn’t get the [prescription] and go to a physical pharmacy for any of these drugs, because they’re specialty drugs—everyone has to really come together. And the coming together doesn’t necessarily always have to happen once a patient is prescribed [a drug]. We need to come together before that and talk about the things that work and don’t work. I hope we can get there sooner rather than later when it comes to solutions. Because if we want to make sure that therapeutic innovation in neurology is really working for everyone, then the conversations need to happen, not when the patient goes to [fill] the drug and asks, “What, prior authorization?” That’s my take; that’s my one hope and wish.